Spontaneous remission in high risk MDS/AML

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biopearl123
Posts: 409
Joined: Fri Jul 20, 2018 5:13 pm

Spontaneous remission in high risk MDS/AML

Post by biopearl123 » Mon Jul 13, 2020 7:52 pm

High risk MDS/AML has a rare spontaneous remission rate. Here is what I found so far:
https://ashpublications.org/bloodadvanc ... -high-risk

They report a 64 year period with 46 cases each a little different. Its pretty rare. Not too sure about lower risk and MF but it makes me wonder if RWD might be applied to further analyze the PII MDS study since there are so many more patients that might be available for a purer "control" matching group. Geron has a highly skilled small group of statistical analysts. Clearly the FDA doesn't care and won't budge failing some catastrophic blood supply shortage, but the more thoughtful and reasonable Europeans might. We know the MDS study has not enrolled to the half way point yet (a promised PR when they do), and I wonder if some of the new sites (where are they by the way) might also be used to provide a RWD look that could be presented to the EU. Continuing to hope for conditional MA in EU but odds are unknown. One hopes reason prevails. It is a terrible thought to think patients will be waiting until 2024 for this drug. Is anyone listening to the KOL's? There is very little downside to a conditional MA while more data is collected. bp

FC4364
Posts: 6
Joined: Sun Dec 23, 2018 11:02 pm

Re: Spontaneous remission in high risk MDS/AML

Post by FC4364 » Tue Jul 14, 2020 4:04 pm

My thoughts are Imetelstat was up for approval with EMA. This would explain the reduction in shorted shares with really no significant price movement. It would also explain, GERN taking on new high profile partners. Then, FDA approved the drug from Astex Pharmaceuticals and this could be part of the reason EMA did not move forward with approval. These are just my thoughts, have no proof, but in a very short window 20 million shares disappeared from being short. That in itself an incredible feat with really no news. Wow!

GERN released that EMA gave them a positive opinion for Orphan Drug designation for Imetelstat which carry's market exclusivity for 10 years. This in itself is significant. Do not know if EMA was just handing GERN a bone to appease them for not approving Imetelstat or if approval will come when new positive data is presented, possibly at ASH or sooner.

At this time, No news is expected before ASH, so no reason for share price to remain where it was....

All in my opinion, of course.

biopearl123
Posts: 409
Joined: Fri Jul 20, 2018 5:13 pm

Re: Spontaneous remission in high risk MDS/AML

Post by biopearl123 » Tue Jul 14, 2020 5:57 pm

FC4364, Having an oral drug approved is definitely an important event but the Astex combination is approved for high risk MDS and CMML, a little bit of a different disease universe. So far I am not sure if there is overlap with the lower risk group that Imetelstat targets. I agree that no clinical news is likely before ASH but we will likely see EU orphan status final approval as noted by Geron by the end of July, and maybe some glimpse as to whether preliminary MA is possible. Also at any time a partner could be announced. We will also see some PR regarding enrolling to the half way point in the MDS trial. If we get a partner, the AML study will likely be resurrected as might the institution of other studies like PV. One would think data already exists for ET approval but we have been over that a lot in the past. So the opportunity for "news" is still out there. As to the shorted shares and what they and their timing means I appreciate your clarification since I have no business background and market machinations/manipulations are way beyond me. Thank you for posting. bp

FC4364
Posts: 6
Joined: Sun Dec 23, 2018 11:02 pm

Re: Spontaneous remission in high risk MDS/AML

Post by FC4364 » Sat Jul 18, 2020 3:55 pm

BP,

Thanks for the clarification on the Astek combination oral solution for MDS/CMML. It is a step forward in helping folks and hopefully it is a positive for the patients.

I still believe Imetelstat was being reviewed for approval by EMA. Just a hunch, no proof but there were interesting events taking place at that time. Imetelstat is the real deal and making folks who need it wait for another 3 to 4 years, well it just does not make any sense.

In regards to the 20 + million shorted shares and there miraculous disappearance from the short's inventory almost seemingly overnight with no significant price movement. I have no information or knowledge as to how the market is manipulated or even if it is. In fact, I am notorious for buying High and selling low.

Thank you again.

FC

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